Chemically modified messenger rna's
AssigneeNovartis AG
InventorsShari Lynn CAPLAN, Katsumasa NAKAJIMA
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Abstract
This invention provides messenger RNA (mRNA) molecules comprising an open reading frame that encodes a protein of interest, wherein said modified RNA comprises a modified nucleoside selected from the group consisting of: (I), (II), and (III), gene therapy vectors comprising same, methods of synthesizing same, and methods for gene replacement, gene therapy, gene transcription silencing, and the delivery of therapeutic proteins to tissue in vivo, comprising the molecules. The present invention also provides methods of reducing the immunogenicity of mRNA molecules.
Claims20
A modified messenger RNA comprising an open reading frame that encodes a protein of interest, wherein said modified RNA comprises a modified nucleoside selected from the group consisting of:
A method for inducing a mammalian cell to produce a protein of interest comprising the step of:
- repeatedly administering to said mammalian cell modified messenger RNA comprising an open reading frame that encodes a protein of interest, wherein said modified messenger RNA comprises at least one modified nucleoside selected from the group consisting of
A method of treating a condition in a mammal caused by a deficiency of a protein of interest, comprising:
- administering a therapeutically effective amount of a modified messenger RNA comprising an open reading frame that encodes the protein of interest, wherein said modified RNA comprises a modified nucleoside selected from the group consisting of:
- wherein said modified messenger RNA is taken up by a cell of said mammal, and said protein of interest is translated from said modified messenger RNA by said mammalian cell;
- thereby relieving said condition in said mammal.
Dependencies are inferred from the claim text.